Posts Tagged ‘medical advances’

After decades of research and testing, the FDA approves belatacept, and a new class of transplant drugs first discovered by Emory doctors.

Back in September, Dr. Christian P. Larsen, Director of the Emory Transplant Center, shared a story with you here on our blog about belatacept, a new medication that was being studied to determine its ability to help block the immune system from graft rejection after kidney transplants. It’s been less than a year since we shared that story on belatacept with you, and since that time, the FDA has now approved belatacept for use for that exact purpose.

Dr. Christian Larsen, Director of Emory Transplant Center

Dr. Thomas Pearson, Surgical Director, Kidney Transplant Program

Since the early 1990s, Emory surgeon-scientists Christian P. Larsen, MD, DPhil and Thomas C. Pearson, MD, DPhil have been searching for ways to promote immune tolerance of a transplanted organ. In collaboration with other Emory researchers and researchers at Bristol-Myers Squibb, they played a leading role in discovering belatacept and driving its development. The recent FDA approval of use of belatacept is the first time a new class of drugs has been developed for transplant since the 1990s.

So what led to this approval of a new class of drugs? From a research perspective, in the 1990s, Larsen and Pearson found that CTLA4-Ig, a fusion protein of which belatacept is a modified type, could control graft rejection in mice, but found that it didn’t work as well in non-human primates. Bristol-Myers Squibb researchers then developed a panel of hundreds of modified forms of CTLA4-Ig, and sifted through the mutated proteins to find two that could make CTLA4-Ig bind tighter to its target and work more effectively. Larsen and Pearson then showed that the enhanced version could prevent graft rejection in a non-human primate model for kidney transplant at Yerkes Research Center.

Once the determination was made that modified versions of the CTLA4-lg fusion proteins could work to prevent graft rejection on primates, belatacept was developed and tested. In two parallel studies with more than 1,200 participants over two years, patients taking belatacept had similar graft survival rates to those taking the calcineurin inhibitor cyclosporine, while maintaining higher kidney function and lower blood pressure and cholesterol. In addition, belatacept can be given every few weeks, in contrast to calcineurin inhibitors, which must be taken twice a day.

There is still room for improvement, though. Compared with cyclosporine-treated patients, belatacept-treated patients had a higher rate of early acute rejection – a temporary flare-up of the immune system against the donated kidney. However, in most cases the acute rejection was successfully treated with drugs and did not lead to graft failure. The Emory Transplant Center team is researching approaches to reduce this risk.

“Our goal is to achieve a normal life span for kidney transplant patients, and have them survive dialysis-free,” Larsen, Director of the Emory Transplant Center, says. “We believe belatacept can help us move toward that goal.”

Clinical trials are now also being conducted to determine if belatacept will have similar positive outcomes on liver transplant and pancreatic islet transplant patients.

For more information on belatacept, you can check out the video below. If you have additional questions, leave them in the comments for Dr. Larsen or Dr. Pearson and we’ll make sure they see them and give you a response!

The Emory Transplant Center is the only transplant center in Atlanta or the state of Georgia, and one of a few places in the country, performing a novel, life-saving protocol to treat bile duct cancer (cholangiocarcinoma). Cholangiocarcinoma is a lethal and aggressive cancer. Traditionally, the disease is treated with resection, surgically removing the tumor, but in many cases the cancer tends to continue to spread around the bile duct. In the past, patients with non-resectable bile duct cancer had little chance of survival.

The new protocol combines chemotherapy and radiation with a liver transplant, improving the likelihood of removing the entire source of cancer during surgery. The chemotherapy and radiation treat and sterilize the tumor bed, but using these options alone may eventually cause liver failure and thus the need for replacing the liver by performing a transplant.

Until recent years, patients diagnosed with cholangiocarcinoma had few treatment options and little chance of survival. This new protocol offers hope and optimism to patients with this difficult disease. Learn more about treatment for bile duct cancer from the video below:

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